Transplant vs Trial vs Ruxolitinib

[sydunipete]

I'm post ET MF, CALR Type 1 and TP53. For 11 years I was on Hydroxyurea (HU) and doing well until 12 months ago when I took a more active interest in my condition, changed specialists and had my first BMB. About 5 months ago I changed from HU to Ruxolitinib and my dose quickly changed from 15mg twice a day to 20 and now 25 which is the maximum dose. My platelets are still in the 700's.

My specialist referred me to a transplant team for consideration of a stem cell transplant, I had a very long and informative chat with Warwick (many thanks mate) and others. I'm still progressing that option as a last resort fall-back.

Concurrently I investigated the CALR antibody trials. I believe there are 2 - one by Incyte (discussed elsewhere in these forums) and another by Janssen (which I don't believe is available in Australia). I went to Adelaide to meet with Dr David Ross. The aim was to gather my thoughts about transplant vs trial vs staying with existing medication.

The meeting with Dr Ross was well worth the plane fare. He's a lovely chap, very smart, very caring and with good knowledge of this space. He managed to explain some things that hadn't been clear to me in the past - very informative.

Regarding the transplant option: TP53 is particularly hard to kill and has a higher incidence of recurrence. It also has a higher incidence of progressing to AML. Because I have a real mixed heritage that makes finding a suitable donor more difficult (although we are yet to start a search). And I'm 62 years old.

Regarding the trials: The current trial is showing promise and I'm pretty sure he said his first patients on the trial are coming up to 3 years. CALR type 1 seem to do better than CALR Type 2. There is a new trial starting in the middle of this year in Adelaide and other sites yet to be determined. The new trial uses the same CALR antibody with the addition of a bio element that trains your T cells to also fight the cancer. (Pardon me if I'm not expressing this technically correct.)

I didn't think I was eligible for the trails. My spleen is normal size and Dr Ross found that quite puzzling. All documentation I've found says you need to have an enlarged spleen and demonstrate a suboptimal response to Ruxolitinib. Dr Ross said if you are ET you don't need an enlarged spleen so he could recommend me for the trial on that basis. The fact that I'm maxing out on Ruxolinib and have a wealth of side-effects from it mean I meet the second criteria.

The current trial drug involves an infusion every second week (for life), the mid-year trial involves an infusion every 3 weeks. Dr Ross is aware that they are working on a self-injectable version (like Pegasys) and a long-lasting version but those are way in the future.

I was really impressed with the Adelaide facilities. They have a large and modern trials centre next door to a research centre. (In our hotel were a number of guests in town for treatment, so the advanced medicine options available in Adelaide are probably of commercial benefit to the city as well as the patients.)

So I've put my name down for the trial. Once it opens up "in the middle of the year" I will have to do some tests (including another BMB) and if accepted I believe you have a fairly short period of time to commence the trial. I would like for Sydney to be a trial centre but Adelaide isn't the worst possible option. From now I will scale back on Ruxo and return to HU and hopefully feel better as a result. To participate in the trial you need to be Ruxo-free for 3 months.

All-in-all it was a very successful trip, way beyond my expectations, and I'm hopeful of being accepted into the trial. I'll update this forum as things progress.

[MPN-MATE Admin]

Evening Peter...

Thank you so much for making this most informative & enlightening POST concerning the monoclonal antibody CALR Clinical Trials being run by Ass Prof' Dr David Ross in Adelaide.

Naturally, I am certain that everyone would be admiring & praising the work he and his research team have conducted thus far...

Much other newer science may also come to the fore through all of this wonderful science & naturally I look forward to learning much more as things progress...

Interesting discovery too that CALR2 is not as successful, (that's me of course!) However, I shall continue to watch w/ great enthusiasm as those CTs progress to Phase 2 & 3 Human trials.

Thank you so much Peter...

Best wishes

Steve

[Kinsale]

Wow Pete - great post and very informative! I hope going back to HU works for you.
Cheers
Mark